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Genome Engineering Lab

The Genome Engineering Lab, or Gel, has several key projects, all of them involving genome editing mediated by the CRISPR/Cas technology. Discovered just a few years ago, CRISPR/Cas has emerged as one of the hottest scientific trends. This technology is speeding up the approach of a futuristic era when genome modifications will permeate human existence.

At Gel, we seek answers to the how questions:
• How do we edit the genome?
• How do we cure incurable diseases?
• How do we improve our lives? And so on …

Gel focuses on discovering new effectors — molecular scissors, which are a key component of the CRISPR/Cas technology. The reason is that for complex genome editing, we need instruments that are way more sophisticated and specific than what we currently have.

We are trying to implement genome editing technologies for developing two kinds of therapies:
• Firstly, we are working on engineered T cells for cancer treatment, because we believe that the immune system is capable of controlling most oncological states. It just needs a little help.
• Secondly, we are developing a technology for generating hematopoietic stem cells to address the bone marrow transplantation problem.

The global demand on bone marrow transplantations is on the order of 5 million procedures annually. However, fewer than 50,000 transplantations per year are made. Unfortunately, it is not always possible to find a compatible donor, but everyone is fully genetically compatible with themselves. So if it becomes possible to make hematopoietic stem cells from a patient’s own tissue, the donation and compatibility issue will be resolved.

As new effectors are discovered, another part of the team takes over to test and implement them for endowing plants and animals with the necessary properties. For instance, this enables the creation of birds resistant to influenza. These studies are in close contact with the current problems of the biotech, agricultural, and food industries. In the future, they will allow companies to avoid the enormous losses that arise due to diseases caused by viruses.

By extension, genetic modification is a way to address the risk of natural selection producing mutations that could give the virus an opportunity to overcome the interspecies barrier, infecting humans and potentially leading to a pandemic. Given the present-day scale of populations, this is more likely than we would wish.